Destiny Pharma notes FDA statement on their focus to enable new approaches to fight AMR

Brighton, United Kingdom – 14 June 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel anti-microbial drugs, which address the global problem of anti-microbial resistance (AMR), notes the FDA Commissioner, Scott Gottlieb M.D.’s further announcements this week outlining the regulator’s support of new incentives for companies developing novel anti-infectives through both financial reimbursement and further streamlined clinical trial requirements.

Neil Clark, Chief Executive Officer of Destiny Pharma commented:

“We welcome the FDA’s continued support for companies investing in the development of much needed novel anti-infectives to address the global issue of antimicrobial resistance. Destiny Pharma is well positioned to benefit from such initiatives as it continues the clinical development of its lead drug candidate, XF-73, for the prevention of post-surgical infections.”

The full FDA press release is below

June 12th 2018 Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s efforts to foster discovery and development of new tools to fight antimicrobial-resistant infections

The increase in serious antimicrobial drug resistant infections is a critical public health concern and a growing threat to patients. According to our colleagues at the Centers for Disease Control and Prevention, each year in the U.S. at least 2 million people become infected with bacteria that are resistant to antibiotics and 23,000 people die each year as a direct result of these infections.

As more and more bacteria grow resistant to currently available antibiotics, we must tackle the issue on all fronts and seek new approaches to this persistent and potentially deadly problem. This means helping to ensure good antibiotic stewardship and use in appropriate clinical scenarios. It also means spurring the development of new antibiotics, for instance, by pursuing novel incentive models for developers that take into consideration the challenging economic and usage dynamics of these products.

Despite the growing incidence of these resistant strains of bacteria, there has unfortunately been an overall decline in antibiotic drug research driven largely by the significant obstacles to developing innovations in this category. This is especially true when it comes to developing new antibiotics that work through novel mechanisms that can evade existing patterns of resistance.

Consider patients who are hospitalized with life-threatening infections and need immediate treatment, but the severity and symptoms of the acute illness, such as delirium, may make obtaining informed consent from patients and performing other trial enrollment procedures difficult. This is just one example of the many challenges to conducting effective clinical trials in patients with serious bacterial diseases.

Complicating matters further, many patients with serious infections may have already tried several currently available antibiotics before a clinical trial is considered. If patients have already received substantial treatment for their infection before enrolling in a clinical trial, this exposure to other treatments can make it more difficult to isolate the effects of an investigational drug.

These are some of the scientific challenges. But there are also economic impediments.

Developing new drugs is a costly endeavor. But the current reimbursement model, where drugs are reimbursed based on each episode of their use, presents incentives that run contrary to effective stewardship over new antibiotics that might be highly effective against very rare and dangerous pathogens. When such drugs become available, we try to use them sparingly, lest pathogens become over-exposed to a new mechanism of attack and develop resistance to it. So, providers have imposed understandable restrictions on the use of such drugs. While this represents responsible stewardship, it also means that a novel antibiotic may have a very limited market. If product developers know that they will not be able to recoup their investments, there may be reduced incentive to invest the significant money needed to discover and develop such a drug.

The global effort to educate patients and providers about the importance of reducing the unnecessary and over-use of antibiotics is a crucially important means to slowing the rate of resistance. However, we must acknowledge that these essential antimicrobial stewardship programs do have an impact – as they should and aim to do – on the use of antibiotics and thus the amount of product sold. And as consequence, we must find ways to spur development and incentivize innovators. The FDA and other federal agencies are taking new steps to address each of these challenges, including new efforts to address the need for better economic incentives.

One of those steps is implementing the set of special incentives that Congress created for antibacterial and antifungal drugs that treat serious or life-threatening infections. This includes the qualified infectious disease product (QIDP) designation. Under this program, new drug applications that are designated as QIDP can receive fast track designation, priority review designation and a possible five-year extension of any exclusivity that the application qualifies for upon approval.

Even with these incentives, we recognize that challenges remain. As such, we continue to work with Congress, our partners at other agencies and the scientific community to find additional ways to create incentives for the development of novel antimicrobial drugs and strengthen the research and development pipeline.

One idea that we’re currently discussing with other agencies such as the Centers for Medicare and Medicaid Services (CMS) would involve changing the model for reimbursement of certain new, anti-microbial drugs that meet critical, public health needs – principally their ability to effectively target dangerous, multi-drug resistant infections.

Under such an approach, instead of paying for drugs that meet a narrow set of critical, public health criteria on a per use basis – for each prescription that’s written, as is done now – one might move instead to a licensing model. Under such a model, the acute care institutions that are most likely to prescribe these medicines would pay a fixed licensing fee for access to the drug, which would offer them the right to use a certain number of annual doses. This is similar to the way that software often gets reimbursed, where institutions pay a licensing fee for a fixed number of installations. We have been speaking with our counterparts at CMS as to whether such an approach is feasible, whether it can be formulated as a demonstration, and as a demonstration, whether it would have the intended public health benefits.

These concepts are still being developed and we look forward to greater public engagement around these ideas. Adapting this licensing payment model to drugs that target dangerous, antimicrobial resistant organisms can help achieve two important public heath goals. First, such a model would create a natural market for drugs that meet certain public health criteria, by providing a predictable return on investment and revenue stream through more foreseeable licensing fees. Second, it would put the institutions fully in charge of stewardship of these important medicines. Once they purchase the ability to access a drug, they would be stewards of its use up to a certain number of annual doses, which could be tied to the number of beds an institution has or its likelihood of encountering certain organisms.

This reimbursement model would address some of the investment challenges associated with the market for potent antimicrobials that target multidrug resistant organisms. These are drugs that we want to have available to us, but that we should keep in reserve and hope that we seldom have to use them. It is my belief that a licensing model might offer an effective “pull incentive” that attempts to create a predictable market for antimicrobial drugs that would meet a narrow set of critical, public health criteria.

We are currently discussing these ideas as part of the FDA’s broader policy work in this area. We plan to release more information soon. Such an approach potentially de-links the return on investment on an important antimicrobial drug from the volume of that drug that’s used. This would achieve an important public health purpose since these are drugs that we would want to hold in reserve.

We’re also taking other new steps to help advance development of improved antimicrobial drugs, through measures that make the development process more predictable and efficient.

Towards these ends, another new program is the Limited Population Pathway for Antibacterial and Antifungal Drugs, or LPAD pathway; established by Congress under the 21st Century Cures Act. The FDA believes this program will advance development and approval of antibacterial drugs to treat serious or life-threatening infections in limited populations of patients with unmet needs. Today FDA issued a draft guidance to assist in the development of drugs using this additional, important pathway.

This draft guidance, when finalized, will support drug development by describing the criteria, processes and other general considerations for drugs approved under the LPAD pathway. In reviewing an application submitted under the LPAD pathway, the FDA will consider the severity, rarity or prevalence of the infection that the drug is intended to treat. The agency will also consider the availability or lack of alternative treatment in the limited population.

The guidance will also assist companies in developing labeling, including prescribing information, patient labeling and carton/container labeling, to inform the medical community that the drug was approved under the LPAD pathway based on a benefit-risk assessment in a limited population.

We’ve already had meaningful, early interest by innovators in potentially developing drugs under this new pathway. We expect that development programs for drugs eligible for approval under the LPAD pathway will follow streamlined approaches to clinical development. This may involve smaller, shorter or fewer clinical trials. However, the LPAD pathway still requires these drug products meet the FDA’s approval standard for safety and effectiveness. Early and frequent communications between the FDA and drug companies interested in pursuing approval under the LPAD pathway for their products can help reduce overall product development times.

While we hope our work will help advance the development of new antibacterial and antifungal drugs, this isn’t a problem that can be addressed by our agency alone. We’re collaborating with agency partners, the broader scientific and policy community, and medical product sponsors to address scientific challenges. Bacteria will continue to evolve. They will continue to chip away at the usefulness of available treatments – including the medicines that we have relied on for years.

Many of the existing antibiotics are simply old. They were screened out of nature where they resided in soil for centuries, engaging in a natural battle with various bugs.

More judicious use of antibiotics in health care and agriculture settings can help slow the rate at which bacteria become resistant to antibiotics. But even with prudent use, we will need to continuously encourage the development of new therapeutic options to keep pace with these challenges. The steps we’re taking, including the issuance of today’s draft guidance on the LPAD pathway and the discussion of new incentive models with our partners at other agencies, are some of the additional steps we’re taking towards strengthening the fragile antibacterial drug pipeline, as part of the larger effort to combat antibiotic-resistant bacteria.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Scientific Advisory Board

Destiny Pharma has today announced the formation of a new Scientific Advisory Board (SAB) to provide expert, independent analysis of the Company’s research and clinical development plans and to help in developing clinical strategies for its unique XF Platform.

Further details of the SAB can be found here.

Posting of Accounts and Notice of AGM

Destiny Pharma plc
(“Destiny Pharma” or “the Company”)

Posting of Annual Report and Accounts, and Notice of AGM

Brighton, United Kingdom – 23 April 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel anti-microbial drugs, which address the global problem of anti-microbial resistance (AMR), announces that the annual report and accounts of the Company for the year ended 31 December 2017 (“Annual Report”), and the notice of the Company’s Annual General Meeting (“AGM”) are now available on the Company’s website at www.destinypharma.com

The Annual Report will be posted to shareholders today.

The AGM is to be held at 10.00am on 31 May 2018 at the offices of FTI Consulting LLP at 200 Aldersgate, London EC1A 4HD.

Clinical update

THE INFORMATION CONTAINED WITHIN THIS ANNOUNCEMENT IS DEEMED BY THE COMPANY TO CONSTITUTE INSIDE INFORMATION AS STIPULATED UNDER THE EU MARKET ABUSE REGULATION (596/2014). UPON THE PUBLICATION OF THE ANNOUNCEMENT VIA A REGULATORY INFORMATION SERVICE, THIS INFORMATION IS CONSIDERED TO BE IN THE PUBLIC DOMAIN.

Destiny Pharma plc
(“Destiny Pharma” or “the company”)

Destiny Pharma announces update on clinical programme for lead drug candidate XF-73

Started recruiting subjects in latest Phase 1 dermal study under US IND

FDA discussions have clarified overall clinical trial plans for XF-73

Phase 2b study on track to announce results in H2 2019

Brighton, United Kingdom – 12 April 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel anti-microbial drugs, which address the global problem of anti-microbial resistance (AMR), today announces an update on the scope and timing of the XF-73 (exeporfinium chloride) Phase 1 and Phase 2b clinical trial programmes following discussions with the US Food and Drug Administration (FDA). This process is in line with the Company’s strategy set out at the time of the placing and admission to AIM and the current status is summarised below:

  • As previously announced, in February 2018, Destiny Pharma opened an Investigational New Drug (IND) application and, in March 2018, was awarded Fast Track designation for the XF-73
  • Destiny Pharma announces today that a Phase 1 dermal safety study, looking at potential skin irritation of XF-73 solution in up to 30 subjects, has started in a specialist US based unit
  • The FDA has clarified that only one additional Phase 1 dermal safety study,looking at skin irritation of XF-73 nasal gel in up to 30 subjects ,has to be completed before the planned Phase 2b trial can start
  • Destiny Pharma is pleased to note that the FDA confirmed that the additional, larger, Phase 1 dermal safety study, looking at the potential for skin sensitisation of XF-73 nasal gel in 200 subjects, does not need to be completed before the Phase 2b study and is only required in advance of the start of Phase 3 trials. Destiny Pharma plans to complete this larger skin sensitisation study in parallel with the Phase 2b in 2019
  • With these clarifications Destiny Pharma is now finalising plans to start the Phase 2b trial. This trial will assess the efficacy of XF-73 against placebo in the nasal decolonisation of Staphylococcus aureus in approximately 150 patients prior to their undergoing hospital surgical procedures. The Company is on track to announce Phase 2b results in the second half of 2019, with the aim of delivering a Phase 3 ready package by the end of 2019

Neil Clark, Chief Executive Officer of Destiny Pharma, commented:

“We are very pleased to get this confirmation from the FDA on the clinical pathway for our lead asset, XF-73. As we set out at the time of our admission to AIM, once the IND and Fast Track designation were confirmed we were expecting to be able to parallel track some of the standard Phase 1 studies and our Phase 2b study based on the existing safety profile of XF-73, and the FDA has now confirmed that this approach is appropriate. Over 160 subjects to date have been dosed with this potentially breakthrough antibacterial drug as a nasal formulation with no significant issues. The Company is well funded and the team at Destiny Pharma remains fully focused on delivering the agreed clinical plan.”

XF-73, Destiny Pharma’s lead asset, is a first-in-class drug candidate, initially being developed for the prevention of post-surgical staphylococcal infections, such as methicillin-resistant Staphylococcus aureus (MRSA), which cause significant complications and increased healthcare burden in the hospital setting.

 

For further information, please contact:

Destiny Pharma plc
Neil Clark, CEO
Simon Sacerdoti, CFO
pressoffice@destinypharma.com
+44 (0)1273 704 440

FTI Consulting
Simon Conway / Victoria Foster Mitchell
destinypharma@fticonsulting.com
+44 (0) 20 3727 1000

Cantor Fitzgerald Europe (Nominated Adviser and Joint Broker)
Philip Davies / Will Goode, Corporate Finance
Andrew Keith, Healthcare Equity Sales
+44 (0)20 7894 7000

 finnCap Ltd (Joint Broker)
Geoff Nash /Kate Bannatyne, Corporate Finance
Alice Lane, Corporate Broking
+44 (0)20 7220 0500

 

About Destiny Pharma

Destiny Pharma is an established, clinical stage, innovative biotechnology company focused on the development of novel medicines that represent a new approach to the treatment of infectious disease. These potential new medicines are being developed to address the need for new drugs for the prevention and treatment of life-threatening infections caused by antibiotic-resistant bacteria, often referred to as “superbugs”. Tackling anti-microbial resistance has become a global imperative recognised by the WHO and the United Nations, as well as the G7 and the G20 countries. For further information, please visit https://www.destinypharma.com

Forward looking statements

Certain information contained in this announcement, including any information as to the Group’s strategy, plans or future financial or operating performance, constitutes “forward-looking statements”. These forward looking statements may be identified by the use of forward-looking terminology, including the terms “believes”, “estimates”, “anticipates”, “projects”, “expects”, “intends”, “aims”, “plans”, “predicts”, “may”, “will”, “seeks” “could” “targets” “assumes” “positioned” or “should” or, in each case, their negative or other variations or comparable terminology, or by discussions of strategy, plans, objectives, goals, future events or intentions. These forward-looking statements include all matters that are not historical facts. They appear in a number of places throughout this announcement and include statements regarding the intentions, beliefs or current expectations of the Directors concerning, among other things, the Group’s results of operations, financial condition, prospects, growth, strategies and the industries in which the Group operates. The directors of the company believe that the expectations reflected in these statements are reasonable, but may be affected by a number of variables which could cause actual results or trends to differ materially. Each forward-looking statement speaks only as of the date of the particular statement. By their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future or are beyond the Group’s control. Forward looking statements are not guarantees of future performance. Even if the Group’s actual results of operations, financial condition and the development of the industries in which the Group operates are consistent with the forward-looking statements contained in this document, those results or developments may not be indicative of results or developments in subsequent periods.

 

 

Audited results for the year ended 31 December 2017

Destiny Pharma plc
(“Destiny Pharma” or “the Company”)

Audited results for the year ended 31 December 2017

Well funded and on track to deliver Phase 2b headline data in H2 2019 for lead
asset, XF-73,a novel anti-microbial

 Brighton, United Kingdom – 12 April 2018 – Destiny Pharma (AIM: DEST), a clinical stage biotechnology company focused on the development of novel anti-microbial drugs, which address the global problem of anti-microbial resistance (AMR), announces its audited financial results for the year ended 31 December 2017.

Financial highlights

  • Successfully completed placing and admission to AIM, raising £15.3 million
  • Additional £3.0 million invested by China Medical Systems Holdings Ltd
  • Strong cash position with cash and term deposits at 31 December 2017 of £16.7 million (2016: £1.5 million)

Operational highlights

  • China and Asia regional development and collaboration agreement signed with China Medical System Holdings Ltd (CMS)
  • Patent portfolio strengthened with the grant of the South Korean XF-biofilm patent
  • Board and executive management team strengthened with the appointment of Neil Clark as CEO, Dr Bill Love, founder of the Company, as Chief Scientific Officer and Dr Huaizheng Peng, General Manager of International Operations at CMS, as Non-executive Director

Post-period highlights

  • US Investigational New Drug (IND) application opened for XF-73
  • US Food and Drug Administration (FDA) Fast Track designation granted for XF-73
  • First subject enrolled in a Phase 1 dermal safety study for XF-73
  • Clarification, through dialogue with the FDA, of the Phase 1 and Phase 2b clinical trials programme – further details of which can be found in a separate announcement released this morning
  • Patent portfolio expanded with the grant of Canadian XF-biofilm patent

Neil Clark, Chief Executive Officer of Destiny Pharma, commented:

“In 2017, we built on the existing strong foundations of Destiny Pharma, successfully completing the placing admission to AIM which raised significant additional capital to progress our pipeline through the planned clinical studies, and executing a key strategic partnership in Asia with China Medical Systems to help realise our commercial aspirations.

“In 2018, progress has continued to be been strong for our lead candidate, XF-73, with the opening of the IND and award of Fast Track designation. Subsequent discussions with the FDA have clarified the clinical pathway for XF-73. We remain on track to deliver Phase 2b results in H2 2019 as part of a Phase 3 ready package. Importantly, our market analysis continues to support the clinical need and commercial opportunity for XF-73 in the prevention of post-surgery hospital infections, such as MRSA, which is estimated to be over a $1 billion market opportunity.

“The Company is well funded following the placing at the time of admission to AIM and, whilst our main focus is on our lead asset, we are also looking to progress our earlier pipeline and develop our collaboration with China Medical Systems. There is continuing international support for the development of novel anti-infective drugs that address the issue of anti-microbial resistance and Destiny Pharma’s unique platform is very well-positioned to meet this global need.”
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The full text of the announcement may be found here.